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Article

Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation.

Science translational medicine - 10 Jul 2024

Zhu Wenliang, Du Wan, Rameshbabu Arun Prabhu, Armstrong Ariel Miura, Silver Stewart, Kim Yehree, Wei Wei, Shu Yilai, Liu Xuezhong, Lewis Morag A, Steel Karen P, Chen Zheng-Yi

Abstract excerpt

Mutations in microRNA-96 (MIR96) cause autosomal dominant deafness-50 (DFNA50), a form of delayed-onset hearing loss. Genome editing has shown efficacy in hearing recovery through intervention in neonatal mice, yet editing in the adult inner ear is necessary for clinical applications, which has not been done. Here, we developed a genome editing therapy for the MIR96 mutation 14C>A by screening different CRISPR...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 38985856
DOI: 10.1126/scitranslmed.adn0689
PMCID: PMC7616320

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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